Dr. Goker Speaks at WorldSymposium
Monitoring and management of inflammation in Gaucher disease: Challenges, options and useful tools.(Accredited provider: Postgraduate Institute for Medicine (PIM); Jointly provided by PIM and Saterdalen & Associates, LLC; Supported by an independent educational grant from Takeda)
https://sponsor.worldsymposia.org/wp-content/uploads/Takeda-Handout-v1.pdf
A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Children With Gaucher Disease Under 5 Years Old
Study Description
Brief Summary:
The main aim of this study is to learn if velaglucerase alfa (VPRIV) improves growth and symptoms in participants with Gaucher disease that are under 5 years old. Symptoms will be checked with blood tests.
Another aim of the study is to check if there are any side effects from VPRIV.
The study will take place in the United States.
After the study doctor has checked that each child can take part in this study, part of each participant's medical record will be recorded. Then, information from each participant's ongoing treatment with VPRIV, which will follow standard patient care, will be recorded. In this study, standard patient care means that the participants will receive VPRIV as they usually would from their doctor. Also, no extra tests will be carried out.
For more information, go to https://clinicaltrials.gov/ct2/show/NCT04721366?term=NCT04721366&recrs=ab&draw=2&rank=1
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